Modality
mRNA
MOA
RAS(ON)i
Target
TIGIT
Pathway
Amyloid
Hemophilia ARSVPBC
Development Pipeline
Preclinical
~Dec 2009
→ ~Mar 2011
Phase 1
~Jun 2011
→ ~Sep 2012
Phase 2
~Dec 2012
→ ~Mar 2014
Phase 3
~Jun 2014
→ ~Sep 2015
NDA/BLA
~Dec 2015
→ ~Mar 2017
Approved
Jun 2017
→ Nov 2030
ApprovedCurrent
NCT05649699
1,760 pts·Hemophilia A
2022-12→2028-11·Terminated
NCT07839774
915 pts·Hemophilia A
2019-07→2028-09·Recruiting
NCT07230849
2,277 pts·Hemophilia A
2017-11→2026-03·Completed
+1 more trial
6,457 total pts2 indications
CompletedCurrentUpcoming
Catalysts (4)
2026-03-054w agoPh3 Readout· Hemophilia A
2028-09-152.5y awayPh3 Readout· Hemophilia A
2028-11-152.6y awayPh3 Readout· Hemophilia A
2030-11-064.6y awayPh3 Readout· PBC
Trial Timeline
Q2Q3Q42018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q4
Approved
Not yet…
Approved
Complet…
Approved
Recruit…
Approved
Termina…
Catalysts
Ph3 Readout
2026-03-05 · 4w ago
Hemophilia A
Ph3 Readout
2028-09-15 · 2.5y away
Hemophilia A
Ph3 Readout
2028-11-15 · 2.6y away
Hemophilia A
Ph3 Readout
2030-11-06 · 4.6y away
PBC
RecruitingCompletedTerminatedNot yet recruiting|StartCompletionToday
Trials (4)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT05649699 | Approved | Hemophilia A | Terminated | 1760 | Biomarker |
| NCT07839774 | Approved | Hemophilia A | Recruiting | 915 | 6MWD |
| NCT07230849 | Approved | Hemophilia A | Completed | 2277 | BodyWt |
| NCT08590467 | Approved | PBC | Not yet recr... | 1505 | Safety |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Zanuderotide | Johnson & Johnson | Phase 2/3 | TIGIT | |
| MRK-6781 | Merck & Co | Approved | TIGIT | |
| SNY-9073 | Sanofi | Phase 2 | B7-H3 | |
| Pexamavacamten | Daiichi Sankyo | NDA/BLA | TIGIT | |
| Tixarelsin | Vertex Pharma | Phase 1 | ALK | |
| BNT-5232 | BioNTech | NDA/BLA | TIGIT | |
| ALN-8757 | Alnylam | Preclinical | TIGIT | |
| Miritinib | Neurocrine | Phase 3 | TIGIT | |
| Fixatuximab | Legend Biotech | Preclinical | CFTR | |
| ION-1378 | Ionis | NDA/BLA | TROP-2 |